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Penicillamine
go.drugbank.com/drugs/DB00859ApprovedInvestigationalIt is an α-amino acid metabolite of penicillin, although it has no antibiotic properties.
Categories: Compounds used in a research, industrial, or household settingTazemetostat
go.drugbank.com/drugs/DB12887ApprovedInvestigational[L11476] Tazemetostat was first named in literature as EPZ-6438.[A190363] Tazemetaostat was granted FDA approval on 23 January 2020.[L11476]
Atidarsagene autotemcel
go.drugbank.com/drugs/DB17538ApprovedInvestigational[L45191] Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic … [L45196] It was granted FDA approval under the brand name Lenmeldy in March 2024.[L50256]
Synonyms: Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) geneBrepocitinib
go.drugbank.com/drugs/DB15003ApprovedInvestigationalBrepocitinib is an oral Janus kinase (JAK) and tyrosine kinase 2 (TYK2) inhibitor used to treat dermatomyositis in adults. … [L64097] The FDA approved brepocitinib on August 27, 2026 for the treatment of dermatomyositis in adult patients, making it the first targeted oral therapy approved for the condition.[L64115]
Fosdenopterin
go.drugbank.com/drugs/DB16628ApprovedIn 2009 a recombinant, E. coli-produced cPMP was granted orphan drug designation by the FDA, becoming the first therapeutic option for patients with MoCD type A. … Molybdenum cofactor deficiency (MoCD) is an exceptionally rare autosomal recessive disorder resulting in a deficiency of three molybdenum-dependent enzymes: sulfite oxidase (SOX), xanthine dehydrogenase
Nerandomilast
go.drugbank.com/drugs/DB18237ApprovedInvestigational[L54126] On October 7, 2025, nerandomilast was approved as the first new treatment for idiopathic pulmonary fibrosis in more than a decade. … [A274581] On December 19, 2025, the U.S. FDA approved nerandomilast tablets for the treatment of progressive pulmonary fibrosis (PPF) in adults.
Difenoxin
go.drugbank.com/drugs/DB01501ApprovedIllicitDifenoxin is an active metabolite of the anti-diarrheal drug, diphenoxylate, which is also used in combination with atropine in the brand mixture Lomotil(R). … It works mostly in the periphery and activates opioid receptors in the intestine rather than the central nervous system (CNS). [3] Difenoxin is also closely related to loperamide, but unlike loperamide
Suzetrigine
go.drugbank.com/drugs/DB18927ApprovedInvestigational[A264953] On January 30, 2025, suzetrigine was approved by the FDA as a first-in-class non-opioid analgesic.[L52098] … Suzetrigine is a Na<sub>V</sub>1.8 voltage-gated sodium channel blocker with analgesic properties[A264948] Na<sub>V</sub>1.8 is abundantly expressed in peripheral sensory nerves to play a role in pain
Olutasidenib
go.drugbank.com/drugs/DB16267ApprovedInvestigationalin 2-hydroxyglutarate (2-HG), a metabolite that participates in tumerogenesis. … [L44256,L44261] It is indicated for the treatment of relapsed or refractory acute myeloid leukemia (AML) in patients with a susceptible IDH1 mutation as determined by an FDA-approved test.
Benzthiazide
go.drugbank.com/drugs/DB00562ApprovedThey inhibit Na+/Cl- reabsorption from the distal convoluted tubules in the kidneys. Thiazides also cause loss of potassium and an increase in serum uric acid. … Thiazides cause vasodilation by activating calcium-activated potassium channels (large conductance) in vascular smooth muscles and inhibiting various carbonic anhydrases in vascular tissue.
Synonyms: 6-chloro-1,1-dioxo-3-(phenylmethylsulfanylmethyl)-4H-benzo[e][1,2,4]thiadiazine-7-sulfonamide